International experiences in multicriteria decision analysis (MCDA) for evaluating orphan drugs: a scoping review

ABSTRACT

Introduction: Orphan diseases are low-prevalence conditions with chronically debilitating or life-threatening consequences. Their treatments are generally called orphan drugs (OD). Health–technology assessment processes have traditionally considered cost-effectiveness analysis (CEA), when making reimbursement and pricing decisions for health-care plans. Valuing OD with standard CEA raises important issues due to uncertain evidence, inability to meet cost-effectiveness thresholds for reimbursement and high budget impact, among others. Multi-criteria decision analysis (MCDA) allows to overcome these issues and improve the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of OD.

Areas covered: A scoping review was conducted in order to characterize MCDA frameworks for assessing OD and implementation experiences. We reviewed electronic databases (Medline, Embase, Cochrane Library, EBSCO, CINAHL, EconLit, Web of Science, LILACS, Google Scholar) key journals (Orphanet Journal of Rare Diseases and Value in Health) and organization repositories.

Expert opinion: The theoretical framework for MCDA considers areas related to characteristics of orphan diseases and their technologies’ clinical and economic impact. Participation processes are critical in incorporating societal values in weighting different dimensions and constructing decision rules. Local implementation pilots considering different stakeholders are necessary in order to pinpoint specific barriers and opportunities.

KEYWORDS:

• Health technology assessment
• MCDA
• multi-criteria decision analysis
• orphan disease
• orphan drugs
• rare disease
• scoping review

Article highlights

• Multi-criteria decision analysis (MCDA) can potentially provide different ways to evaluate orphan drugs (OD)

• There is extensive available literature regarding both theoretical frameworks and experiences of implementation of MCDA for OD

• Theoretical frameworks have been extensively discussed with some frameworks being consolidated (i.e. EVIDEM)

• Implementation publication generally covers only portions of the process or are proof-of-concept exercises. Inclusion of actual decision-makers seems to be still limited

• More implementation studies are necessary. Many lessons can be learned from the published ones, especially regarding participation processes

Declaration of interest

P Lasalvia, L Prieto, G Romano, N Garzon and D Rosselli work at NeuroEconomix, which received funding for the realization of this study. M Moreno and J Castrillon are currently employed at Novartis in Colombia. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary Material

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Additional information

Funding

This study received funding from Novartis de Colombia.